US pharmaceuticals giant Vertex Pharmaceuticals and biotech company Moderna Therapeutics have announced an exclusive research collaboration and licensing agreement to discover and develop messenger ribonucleic acid (mRNA) therapeutics for cystic fibrosis (CF).
The three-year collaboration will focus on the use of mRNA therapies to treat the underlying cause of CF by enabling cells in the lungs to produce functional copies of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is known to be defective in people with CF.
“Moderna’s mRNA Therapeutics represent a promising new approach that could provide functional CFTR proteins to the lungs of people with CF, which would complement our existing approach of using CFTR modulators to treat CF,” said Dr. David Altshuler, Vertex’s executive vice president of Global Research and chief scientific officer in a press release.
Moderna CEO Stephen Bancel said he was excited to partner with Vertex as the company had “deep scientific expertise” in regards to creating quality cystic fibrosis treatment regimes.
“We’re particularly excited to explore the potential of pulmonary delivery as a new modality to bring transformative mRNA medicines to patients and address unmet needs,” he said.
mRNA is responsible for carrying genetic instructions transcribed from DNA, which cells then translate to produce proteins that, when defective or missing, can underlie certain diseases, including CF. Moderna’s pioneering mRNA Therapeutics™ are designed to trigger the cellular machinery to utilize their natural processes to produce specific functional proteins. Through this collaboration, mRNA Therapeutics may be developed to enable the delivery of correct genetic instructions into cells in the lungs, which may trigger the cells to produce functional CTFR protein.
You can read Vertex Pharmaceuticals’ press release here.